Global Genetic Disorders Drugs Market to 2023-A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease

(PharmaNewsWire.Com, March 07, 2018 ) The genetic disorders therapy area consists of indications that arise because of an abnormality in a person's DNA. This report focuses on the key therapy area indications of cystic fibrosis (CF), Duchenne muscular dystrophy (DMD), lysosomal storage disease (LSD) and paroxysmal nocturnal hemoglobinuria (PNH).

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Genetic disorders are frequently inherited from an individual's parents but can also arise because of a new (de novo) mutation. The treatment landscape for genetic disorders is dominated commercially by orphan drugs for the treatment of rare genetic disorders.

Overall, 1,390 products are being actively developed in the genetic disorders pipeline. The pipelines of CF, DMD, LSD and PNH contain 163, 109, 174 and 24 products, respectively. The pipeline is dominated by targeted therapies that aim to treat the underlying cause, which is often specific to the particular indication.

Scope
-Global revenue from the genetic disorders market is forecast to increase from $19.6 billion in 2016 to $47.7 billion in 2023, at a compound annual growth rate of 13.55%. What is driving this growth-
-The leading companies in terms of market share are Alexion, Sanofi, Shire and Vertex. Which of these are forecast to experience the largest growth-
-There are new approvals and late-stage products set to enter the market during the forecast period. Which drugs will achieve blockbuster status-
-The market has been dominated by complement system inhibitors and enzyme replacement therapies. To what extent will these classes of drugs and others dominate the market over the forecast period-
-There are 1,390 genetic disorders products in the pipeline. What molecular targets are most abundant in the pipeline and what role will pipeline product approvals play in market growth-
-Genetic disorders clinical trials have an overall attrition rate of around 93%, what can companies do to maximize their chance of success-

Reasons to buy
-Understand the current treatment landscape, with portfolios of key marketed products and a focus on historical and forecast sales patterns, and an overview of each drug's mechanism of action.
-Analyze the genetic disorders pipeline through a comprehensive review of the pipeline segmented by stage of development, molecule type and molecular target. This review also provides a detailed look at products for the treatment of genetic disorders to provide an insight into the risk associated with attempting to bring pipeline products to market.
-Predict growth in market size, with in-depth market forecasting from 2016 to 2023. The forecasts will provide an understanding of how epidemiology trends, new drug entries, and patent expirations will influence market value.
-Identify commercial opportunities in the genetic disorders deals landscape by analyzing trends in licensing and co-development deals.

Complete Report is Available at http://www.reportsweb.com/global-genetic-disorders-drugs-market-to-2023

Table of Contents
1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 7
1.2 List of Figures 8
2 Introduction 11
2.1.1 Cystic Fibrosis 12
2.1.2 Duchenne Muscular Dystrophy 12
2.1.3 Lysosomal Storage Disease 12
2.1.4 Paroxysmal Nocturnal Hemoglobinuria 14
2.2 Symptoms 14
2.3 Diagnosis 15
2.3.1 Cystic Fibrosis 17
2.3.2 Duchenne Muscular Dystrophy 18
2.3.3 Lysosomal Storage Disease 19
2.3.4 Paroxysmal Nocturnal Hemoglobinuria 19
2.4 Etiology and Pathophysiology 20
2.4.1 Cystic Fibrosis 20
2.4.2 Duchenne Muscular Dystrophy 21
2.4.3 Lysosomal Storage Disease 22
2.4.4 Paroxysmal Nocturnal Hemoglobinuria 25
2.5 Epidemiology Patterns and Forecasts-Prevalence and Patient Segmentation 26
2.5.1 Cystic Fibrosis 27
2.5.2 Duchenne Muscular Dystrophy 28
2.5.3 Lysosomal Storage Disease 28
2.5.4 Paroxysmal Nocturnal Hemoglobinuria 29
2.6 Co-morbidities and Complications 30
2.6.1 Cystic Fibrosis 30
2.6.2 Duchenne Muscular Dystrophy 31
2.6.3 Lysosomal Storage Disease 31
2.6.4 Paroxysmal Nocturnal Hemoglobinuria 32
2.7 Prognosis 33
2.7.1 Cystic Fibrosis 33
2.7.2 Duchenne Muscular Dystrophy 34
2.7.3 Lysosomal Storage Disease 35
2.7.4 Paroxysmal Nocturnal Hemoglobinuria 37
2.8 Treatment 37
2.8.1 Cystic Fibrosis 37
2.8.2 Duchenne Muscular Dystrophy 39
2.8.3 Lysosomal Storage Disease 41
2.8.4 Paroxysmal Nocturnal Hemoglobinuria 42
3 Key Marketed Products 44
3.1 Overview 44
3.2 Soliris (eculizumab)-Alexion 44
3.3 Orkambi (lumacaftor/ivacaftor)-Vertex 46
3.4 Cerezyme (imiglucerase)-Sanofi Genzyme 47
3.5 Myozyme/Lumizyme (alglucosidase alfa)-Sanofi Genzyme 49
3.6 Fabrazyme (agalsidase beta)-Sanofi Genzyme 51
3.7 Pulmozyme (dornase alfa)-Roche 53
3.8 Kalydeco (ivacaftor)-Vertex 54
3.9 Elaprase (idursulfase)-Shire 56
3.10 Vimizim (elosulfase alfa)-Biomarin 58
3.11 Cerdelga (eliglustat tartrate)-Sanofi Genzyme 60
3.12 Exondys 51 (eterplirsen)-Sarepta 61
3.13 Conclusion 62
4 Pipeline Landscape Assessment 63
4.1 Overview 63
4.2 Pipeline Development Landscape 63
4.3 Molecule Types in the Pipeline 66
4.4 Molecular Targets in the Pipeline 67
4.5 Clinical Trials Landscape 69
4.5.1 Clinical Trial Failure Rates 69
4.5.2 Clinical Trial Duration 73
4.5.3 Clinical Trial Size 77
4.5.4 Cumulative Clinical Trial Size 81
4.6 Assessment of Key Pipeline Products 84
4.6.1 Epidiolex (cannabidiol)-GW Pharmaceuticals 84
4.6.2 ALXN-1210 (ravulizumab)-Alexion 86
4.6.3 Lanadelumab-Shire 87
4.6.4 Burosumab-Ultragenyx and Kyowa Hakko Kirin 88
4.6.5 GBT440 (voxelotor)-Global Blood Therapeutics 90
4.6.6 Ivacaftor and Tezacaftor-Vertex 91
5 Multi-scenario Market Forecast to 2023 93
5.1 Overall Market Size 93
5.2 Generic Penetration 95
5.3 Revenue Forecast by Molecular Target 97
5.3.1 Immune/Inflammatory Mediators 97
5.3.2 Enzyme Replacement/Modifying Therapies 98
5.3.3 Ion Channel Modulators 99
5.3.4 G Protein-Coupled Receptor Modulators 100
5.3.5 Metabolic Enzyme Mediators 101
6 Company Analysis and Positioning 102
6.1 Revenue and Market Share Analysis by Company 104
6.1.1 Alexion-How will Alexion overcome the threat posed by the emergence of Soliris biosimilars-108
6.1.2 Sanofi-How will revenue be affected by the expiry of key patents-109
6.1.3 Vertex-Will Vertex continue to focus heavily on the treatment of CF-110
6.1.4 Shire-How will late-stage pipeline drug Lanadelumab affect Shire's revenue-111
6.1.5 Amgen-Will Amgen develop more products for genetic diseases following the success of Repatha-112
6.1.6 GW Pharmaceuticals-How will Epidiolex drive sales for GW-113
6.1.7 Pfizer-How will Pfizer's small genetic disorders pipeline affect long-term sales-114
6.1.8 BioMarin-To what extent will BioMarin continue to focus exclusively on the treatment of genetic disorders-115
6.1.9 Sarepta-Will Sarepta emerge as a key player in the genetic disorders landscape-116
6.2 Company Landscape 117
6.3 Marketed and Pipeline Portfolio Analysis 118
7 Strategic Consolidations 121
7.1 Licensing Deals 121
7.1.1 Deals by Region, Year and Value 121
7.1.2 Deals by Stage of Development and Value 124
7.1.3 Deals by Molecule Type, Molecular Target and Value 125
7.1.4 Table for Licensing Deals with Disclosed Values 126
7.2 Co-development Deals 130
7.2.1 Deals by Region, Year and Value 130
7.2.2 Deals by Stage of Development and Value 132
7.2.3 Deals by Molecule Type, Molecular Target and Value 133
7.2.4 Table for Co-development Deals with Disclosed Values 134
8 Appendix 138
8.1 References 138
8.2 Figures of All Clinical Stage Pipeline Products 147
8.3 Abbreviations 156
8.4 Disease List 157
8.5 Methodology 158
8.5.1 Coverage 158
8.5.2 Secondary Research 158
8.5.3 Market Size and Revenue Forecasts 159
8.5.4 Pipeline Analysis 159
8.5.5 Competitive Landscape 160
8.6 Contact Us 160
8.7 Disclaimer 160

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Global Genetic Disorders Drugs Market to 2023-A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease